title: Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients.
abstract: Our study was designed to prospectively determine whether or not unrelated cord blood transplantation (CBT) can produce outcomes comparable to related donor transplantation for children with β-thalassaemia. In 35 patients, 40 transplants were performed between October 2003 and September 2009. HLA matching at enrolment was 6/6 (n=8), 5/6 (n=16), 4/6 (n=27), or 3/6 (n=1) by low-resolution HLA-A, -B, and high-resolution DRB1. These patients received non-manipulated grafts without ex vivo expansion or T-cell depletion. The median number of nucleated and CD34+ cells infused was 7.8 × 10(7)/kg (range, 2.8-14.7 × 10(7)/kg) and 4.0 × 10(5)/kg (range, 1.7-19.9 × 10(5)/kg), respectively. The 5-year OS and thalassaemia-free survival after the first transplant were 88.3 and 73.9%, respectively. The cumulative incidence of TRM at 2 years was 11.7%. Fourteen patients developed chronic skin GVHD. Thirty patients were alive and transfusion-independent with a Lansky performance score ≥80% achieved between 6 and 76 months post transplant (median, 36 months). These data compare acceptably with the survival rates of related-donor BMT for thalassaemia and suggest that patients without an available HLA-compatible sibling but who have well-matched unrelated donors should also be considered for CBT.
authors.affiliation: Division of Hematology/Oncology, Department of Pediatrics, Chang Gung Children's Hospital, Chang Gung University, Taoyuan, Taiwan. jaing001@cgmh.org.tw
authors.forename: T-H
authors.initials: TH
authors.lastname: Jaing
authors.forename: I-J
authors.initials: IJ
authors.lastname: Hung
authors.forename: C-P
authors.initials: CP
authors.lastname: Yang
authors.forename: S-H
authors.initials: SH
authors.lastname: Chen
authors.forename: H-T
authors.initials: HT
authors.lastname: Chung
authors.forename: P-K
authors.initials: PK
authors.lastname: Tsay
authors.forename: Y-C
authors.initials: YC
authors.lastname: Wen
mesh_terms: D000293:Adolescent; D018952:Antigens, CD34 / Q000097:blood; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D018572:Disease-Free Survival; D005260:Female; D006680:HLA Antigens; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008212:Lymphocyte Depletion / Q000379:methods; D008297:Male; D011446:Prospective Studies; D012189:Retrospective Studies; D015996:Survival Rate; D013789:Thalassemia / Q000097:blood / Q000401:mortality* / Q000628:therapy*; D014184:Transplantation, Homologous
chemical_list: D018952:Antigens, CD34; D006680:HLA Antigens